Vismodegib holds therapeutic potential in advanced basal cell carcinoma

The Hedgehog pathway inhibitor vismodegib appears to be effective in the treatment of locally advanced periocular basal cell carcinoma (BCC), with adverse events (AEs) that are mostly manageable, as reported in a recent study.

The researchers reviewed the medical records of 21 patients (median age, 76 years; 76.2 percent male) with locally advanced and metastatic orbital (n=15) or periocular (n=6) BCC treated with vismodegib. Treatment went on for a median of 9 months.

Follow-up duration was 26 months overall and 17 months after cessation of treatment. Complete response was seen in 10 patients and partial response in another 10 patients, whereas disease remained stable in the remaining patient. Among the complete responders, five sustained a complete response at 16 months and three who stopped treatment experienced recurrence 8 months later.

Most treatment-related adverse reactions were low grade (grade 1 or 2). Frequently reported grade 1 or 2 complications were muscle spasm (76 percent), dysgeusia (57 percent), alopaecia (47 percent), weight loss (47 percent) and reduced appetite (19 percent). Hepatoxicity was the only documented high-grade (grade 3 or 4) adverse event (10 percent).

Treatment was withdrawn in eight patients due to side effects. There were five deaths overall, most of which were deemed unrelated to vismodegib therapy, except for one patient who died from potentially treatment-related sepsis (grade 5 adverse event).

The present data show that long periods of complete tumour regression can be achieved in some patients even after discontinuation of the medication, according to the researchers. However, the optimal treatment protocol and duration have yet to be established, highlighting a need for more prospective multicenter trials.